Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice

Many inborn errors of metabolism require life-long treatments and, in severe conditions involving the liver, organ transplantation remains the only curative treatment. Non-integrative AAV-mediated gene therapy has shown efficacy in adult patients. However, treatment in pediatric or juvenile settings...

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Detalles Bibliográficos
Autores principales: Lisjak, Michela, De Caneva, Alessia, Marais, Thibaut, Barbon, Elena, Biferi, Maria Grazia, Porro, Fabiola, Barzel, Adi, Zentilin, Lorena, Kay, Mark A., Mingozzi, Federico, Muro, Andrés F.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Genome Editing
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8962648/
https://www.ncbi.nlm.nih.gov/pubmed/35359664
http://dx.doi.org/10.3389/fgeed.2022.785698

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